An observational study in Retinitis Pigmentosa, Retinitis and Retinal Diseases, sponsored by Nanoscope Therapeutics Inc.. Active, not recruiting at 6 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-03-24.
Sponsored by Nanoscope Therapeutics Inc. · Observational
This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines. Eligible subjects will be consented to return for scheduled study visits for this study following their completion in study NTXMCO-002 (RESTORE). They will not receive a second treatment with MCO-010 (or a repeated sham injection) in this study
This study is designed to follow subjects with Retinitis Pigmentosa (RP) previously enrolled in study NTXMCO-002 (RESTORE, NCT04945772). In that study, 18 of 27 enrolled subjects received MCO-010, an ambient light-activated, Multi-Characteristic Opsin (MCO) transgene in an adeno-associated virus serotype 2 (AAV2) vector via intravitreal injection (IVT) and 9 of 27 received a sham injection. Those who received the sham injection will not be continued in the long-term, follow-up study for drug safety. MCO-010 has the potential to restore vision irrespective of the underlying gene mutation, and because it is directed at bipolar retinal cells, intact photoreceptors are not required. Further details on MCO-010 and the underlying disease under investigation are included in the protocol for RESTORE and are not repeated herein.
The current study is a non-interventional long-term safety follow-up of the subjects who completed RESTORE, in accordance with FDA guidance on recipients of human gene therapy products.
278 studies on the registry are indexed under Retinitis Pigmentosa; 72 are open to participants now.
This study's planned enrollment of 18 is below the median of 52 across 87 observational studies indexed under Retinitis Pigmentosa.
Browse Retinitis Pigmentosa studies →Nanoscope Therapeutics Inc. is the lead sponsor of 8 studies on the registry; 2 are open to participants now.
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This study population will be comprised of the 18 adult subjects with advanced retinitis pigmentosa, previously dosed with MCO-010 in the RESTORE study.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
This is a long-term follow-up observational study of participants who previously received 1.2E11gc/eye of MCO-010. No investigational product will be administered in this study.
Biological: Gene Therapy product-MCO-010
This is a long-term follow-up observational study of participants who previously received 0.9E11gc/eye of MCO-010 No investigational product will be administered in this study.
Biological: Gene Therapy product-MCO-010
Safety evaluation to monitor long term effects of previously injected MCO-010 in RP patients
Assessment of the long-term safety of previous treatment with a single intravitreal injection of MCO-010
Delayed adverse events. Incidence, nature, and severity of selected adverse events (AEs); all serious adverse events (SAEs); all ocular AEs including intraocular inflammation graded through ocular exam; non-ocular AEs with a common terminology criteria for adverse events (CTCAE) grade of 3 or greater; AEs of special interest (AESIs) including new malignancies, new incidence or exacerbation of any pre-existing neurologic disorder or rheumatologic or other autoimmune disorder, new incidence of hematologic disorder or new infection regardless of suspected relatedness to treatment with MCO-010.
Time frame: 156 weeks
Evaluation of long-term effects on visual acuity of previous treatment with a single intravitreal injection of MCO-010
Change from baseline in BCVA over time in both eyes
Time frame: 156 Weeks
Evaluation of long-term effects on shape discrimination at multiple light levels of previous treatment with a single intravitreal injection with MCO-010
Change from baseline in multi-luminance shape discrimination test (MLSDT) scores
Time frame: 156 Weeks
Evaluation of long-term effects on navigation/mobility at multiple light levels of previous treatment with a single intravitreal injection with MCO-010
Change from baseline in multi-luminance Y-Mobility Test (MLYMT) score
Time frame: 156 Weeks
Exploration of the long-term impact of previous treatment with MCO-010 on retinal thickness and retinal anatomy
Assessment of fundus photography and Optical Coherence Tomography (OCT) outcomes over time
Time frame: 156 Weeks
Assessment of the long-term pharmacokinetic (PK) and pharmacodynamic (PD) impact of previous treatment with MCO-010 on gene reporter expression
PK parameters including change from baseline of fundus fluorescence intensity of reporter over time and PD correlation of gene expression with the efficacy measures in the study eye and fellow eye (selected sites)
Time frame: 156 Weeks
Plan to share: Yes — The results of the clinical trial will be made available when the study is completed and results are analyzed. The results will be published on this site and be available to conference presentations and publications.
Time frame: Within a year from the long term monitoring data availability
Access criteria: IPD sharing access will be subject to data transfer agreement. IPD generated as part of this clinical study may be subject to patient confidentiality.
No publications or documents are linked to this record.
This study is active, not recruiting, as verified in Mar 2025. You cannot join it, but the record below documents what was studied.
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Nanoscope Therapeutics Inc.